JP4888876B2 - アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター - Google Patents

アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター Download PDF

Info

Publication number: JP4888876B2
Authority: JP; Japan
Prior art keywords: associated virus; adeno; seq; pharmaceutical composition; virus vector
Prior art date: 2003-06-13
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.): Expired - Lifetime

Application number

JP2003371103A

Other languages

English (en)

Japanese (ja)

Other versions

JP2005021149A (ja

Inventor

平武田

英夫原

Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)

Individual

Original Assignee

Individual

Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)

2003-06-13

Filing date

2003-10-30

Publication date

2012-02-29

2003-10-30 Priority to JP2003371103A priority Critical patent/JP4888876B2/ja

2003-10-30 Application filed by Individual filed Critical Individual

2004-06-11 Priority to RU2006101149/13A priority patent/RU2335542C2/ru

2004-06-11 Priority to AU2004248014A priority patent/AU2004248014B2/en

2004-06-11 Priority to AT04745818T priority patent/ATE466093T1/de

2004-06-11 Priority to EP04745818A priority patent/EP1634956B1/en

2004-06-11 Priority to CA2529179A priority patent/CA2529179C/en

2004-06-11 Priority to DE602004026867T priority patent/DE602004026867D1/de

2004-06-11 Priority to ES04745818T priority patent/ES2345151T3/es

2004-06-11 Priority to BRPI0411321-7A priority patent/BRPI0411321A/pt

2004-06-11 Priority to NZ544554A priority patent/NZ544554A/en

2004-06-11 Priority to PCT/JP2004/008224 priority patent/WO2004111250A1/ja

2004-06-11 Priority to KR1020057023896A priority patent/KR20060029225A/ko

2004-06-11 Priority to US10/560,280 priority patent/US8318687B2/en

2004-06-11 Priority to DK04745818.7T priority patent/DK1634956T3/da

2004-06-11 Priority to PT04745818T priority patent/PT1634956E/pt

2005-01-27 Publication of JP2005021149A publication Critical patent/JP2005021149A/ja

2012-02-29 Application granted granted Critical

2012-02-29 Publication of JP4888876B2 publication Critical patent/JP4888876B2/ja

2023-10-30 Anticipated expiration legal-status Critical

Status Expired - Lifetime legal-status Critical Current

Links

241000702421 Dependoparvovirus Species 0.000 title claims abstract description 63
239000013598 vector Substances 0.000 title claims abstract description 50
208000024827 Alzheimer disease Diseases 0.000 title claims description 22
150000001413 amino acids Chemical class 0.000 claims description 32
DZHSAHHDTRWUTF-SIQRNXPUSA-N amyloid-beta polypeptide 42 Chemical compound C([C@@H](C(=O)N[C@@H](C)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@H](C(=O)NCC(=O)N[C@@H](CO)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CCCCN)C(=O)NCC(=O)N[C@@H](C)C(=O)N[C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCSC)C(=O)N[C@@H](C(C)C)C(=O)NCC(=O)NCC(=O)N[C@@H](C(C)C)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](C)C(O)=O)[C@@H](C)CC)C(C)C)NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@@H](NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@H](CC=1N=CNC=1)NC(=O)[C@H](CC=1N=CNC=1)NC(=O)[C@@H](NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)CNC(=O)[C@H](CO)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC=1N=CNC=1)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](C)NC(=O)[C@@H](N)CC(O)=O)C(C)C)C(C)C)C1=CC=CC=C1 DZHSAHHDTRWUTF-SIQRNXPUSA-N 0.000 claims description 32
108010076504 Protein Sorting Signals Proteins 0.000 claims description 29
239000002773 nucleotide Substances 0.000 claims description 28
125000003729 nucleotide group Chemical group 0.000 claims description 28
101710137189 Amyloid-beta A4 protein Proteins 0.000 claims description 20
101710151993 Amyloid-beta precursor protein Proteins 0.000 claims description 20
102100022704 Amyloid-beta precursor protein Human genes 0.000 claims description 20
239000008194 pharmaceutical composition Substances 0.000 claims description 20
FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 5
230000000968 intestinal effect Effects 0.000 claims description 3
230000003248 secreting effect Effects 0.000 claims description 3
108010033276 Peptide Fragments Proteins 0.000 abstract description 21
102000007079 Peptide Fragments Human genes 0.000 abstract description 21
108090000765 processed proteins & peptides Proteins 0.000 abstract description 13
230000004727 humoral immunity Effects 0.000 abstract description 7
230000006698 induction Effects 0.000 abstract description 6
108020004414 DNA Proteins 0.000 description 27
238000012360 testing method Methods 0.000 description 25
241000699666 Mus <mouse, genus> Species 0.000 description 20
239000000427 antigen Substances 0.000 description 20
108091007433 antigens Proteins 0.000 description 20
102000036639 antigens Human genes 0.000 description 20
210000004027 cell Anatomy 0.000 description 17
210000001519 tissue Anatomy 0.000 description 16
210000002966 serum Anatomy 0.000 description 15
241000699670 Mus sp. Species 0.000 description 14
208000037259 Amyloid Plaque Diseases 0.000 description 13
102000046299 Transforming Growth Factor beta1 Human genes 0.000 description 13
101800002279 Transforming growth factor beta-1 Proteins 0.000 description 13
239000002299 complementary DNA Substances 0.000 description 13
108090000623 proteins and genes Proteins 0.000 description 12
229940099456 transforming growth factor beta 1 Drugs 0.000 description 11
238000000034 method Methods 0.000 description 10
239000013612 plasmid Substances 0.000 description 10
230000003941 amyloidogenesis Effects 0.000 description 9
210000004556 brain Anatomy 0.000 description 9
230000007082 Aβ accumulation Effects 0.000 description 8
238000006243 chemical reaction Methods 0.000 description 8
239000000243 solution Substances 0.000 description 8
230000002829 reductive effect Effects 0.000 description 7
230000003612 virological effect Effects 0.000 description 7
241000699660 Mus musculus Species 0.000 description 6
238000004458 analytical method Methods 0.000 description 6
102000004169 proteins and genes Human genes 0.000 description 6
238000011160 research Methods 0.000 description 6
238000011830 transgenic mouse model Methods 0.000 description 6
DGVVWUTYPXICAM-UHFFFAOYSA-N β‐Mercaptoethanol Chemical compound OCCS DGVVWUTYPXICAM-UHFFFAOYSA-N 0.000 description 6
230000002776 aggregation Effects 0.000 description 5
238000004220 aggregation Methods 0.000 description 5
108010064539 amyloid beta-protein (1-42) Proteins 0.000 description 5
238000001415 gene therapy Methods 0.000 description 5
230000016784 immunoglobulin production Effects 0.000 description 5
238000010186 staining Methods 0.000 description 5
238000000692 Student's t-test Methods 0.000 description 4
238000000540 analysis of variance Methods 0.000 description 4
210000001130 astrocyte Anatomy 0.000 description 4
239000000872 buffer Substances 0.000 description 4
230000004663 cell proliferation Effects 0.000 description 4
210000003169 central nervous system Anatomy 0.000 description 4
230000007850 degeneration Effects 0.000 description 4
108020001507 fusion proteins Proteins 0.000 description 4
102000037865 fusion proteins Human genes 0.000 description 4
238000005259 measurement Methods 0.000 description 4
239000000047 product Substances 0.000 description 4
108010090849 Amyloid beta-Peptides Proteins 0.000 description 3
102000013455 Amyloid beta-Peptides Human genes 0.000 description 3
206010012289 Dementia Diseases 0.000 description 3
238000002965 ELISA Methods 0.000 description 3
241001465754 Metazoa Species 0.000 description 3
102000003992 Peroxidases Human genes 0.000 description 3
208000034953 Twin anemia-polycythemia sequence Diseases 0.000 description 3
241000700605 Viruses Species 0.000 description 3
108010064397 amyloid beta-protein (1-40) Proteins 0.000 description 3
210000004204 blood vessel Anatomy 0.000 description 3
230000037396 body weight Effects 0.000 description 3
230000008021 deposition Effects 0.000 description 3
239000002552 dosage form Substances 0.000 description 3
239000003814 drug Substances 0.000 description 3
230000000694 effects Effects 0.000 description 3
238000002474 experimental method Methods 0.000 description 3
239000012634 fragment Substances 0.000 description 3
210000005153 frontal cortex Anatomy 0.000 description 3
230000002496 gastric effect Effects 0.000 description 3
210000001320 hippocampus Anatomy 0.000 description 3
238000000338 in vitro Methods 0.000 description 3
238000002347 injection Methods 0.000 description 3
239000007924 injection Substances 0.000 description 3
210000000274 microglia Anatomy 0.000 description 3
239000000203 mixture Substances 0.000 description 3
210000002569 neuron Anatomy 0.000 description 3
210000001152 parietal lobe Anatomy 0.000 description 3
239000002245 particle Substances 0.000 description 3
108040007629 peroxidase activity proteins Proteins 0.000 description 3
230000002441 reversible effect Effects 0.000 description 3
230000007507 senile plaque formation Effects 0.000 description 3
208000022288 senile plaque formation Diseases 0.000 description 3
125000003831 tetrazolyl group Chemical group 0.000 description 3
229940124597 therapeutic agent Drugs 0.000 description 3
LOGFVTREOLYCPF-KXNHARMFSA-N (2s,3r)-2-[[(2r)-1-[(2s)-2,6-diaminohexanoyl]pyrrolidine-2-carbonyl]amino]-3-hydroxybutanoic acid Chemical compound C[C@@H](O)[C@@H](C(O)=O)NC(=O)[C@H]1CCCN1C(=O)[C@@H](N)CCCCN LOGFVTREOLYCPF-KXNHARMFSA-N 0.000 description 2
108010048112 Amyloidogenic Proteins Proteins 0.000 description 2
102000009091 Amyloidogenic Proteins Human genes 0.000 description 2
HEDRZPFGACZZDS-UHFFFAOYSA-N Chloroform Chemical compound ClC(Cl)Cl HEDRZPFGACZZDS-UHFFFAOYSA-N 0.000 description 2
102000004127 Cytokines Human genes 0.000 description 2
108090000695 Cytokines Proteins 0.000 description 2
101000823051 Homo sapiens Amyloid-beta precursor protein Proteins 0.000 description 2
102000003777 Interleukin-1 beta Human genes 0.000 description 2
108090000193 Interleukin-1 beta Proteins 0.000 description 2
108091034117 Oligonucleotide Proteins 0.000 description 2
108700019146 Transgenes Proteins 0.000 description 2
108060008682 Tumor Necrosis Factor Proteins 0.000 description 2
102000000852 Tumor Necrosis Factor-alpha Human genes 0.000 description 2
238000002835 absorbance Methods 0.000 description 2
230000006933 amyloid-beta aggregation Effects 0.000 description 2
230000003942 amyloidogenic effect Effects 0.000 description 2
230000000692 anti-sense effect Effects 0.000 description 2
230000000890 antigenic effect Effects 0.000 description 2
230000015572 biosynthetic process Effects 0.000 description 2
210000004369 blood Anatomy 0.000 description 2
239000008280 blood Substances 0.000 description 2
210000005013 brain tissue Anatomy 0.000 description 2
AIYUHDOJVYHVIT-UHFFFAOYSA-M caesium chloride Chemical compound [Cl-].[Cs+] AIYUHDOJVYHVIT-UHFFFAOYSA-M 0.000 description 2
230000007969 cellular immunity Effects 0.000 description 2
230000002490 cerebral effect Effects 0.000 description 2
230000001684 chronic effect Effects 0.000 description 2
238000010276 construction Methods 0.000 description 2
238000001514 detection method Methods 0.000 description 2
238000010790 dilution Methods 0.000 description 2
239000012895 dilution Substances 0.000 description 2
206010014599 encephalitis Diseases 0.000 description 2
239000003623 enhancer Substances 0.000 description 2
239000005090 green fluorescent protein Substances 0.000 description 2
230000002757 inflammatory effect Effects 0.000 description 2
210000003734 kidney Anatomy 0.000 description 2
238000004519 manufacturing process Methods 0.000 description 2
239000003550 marker Substances 0.000 description 2
BDAGIHXWWSANSR-UHFFFAOYSA-N methanoic acid Natural products OC=O BDAGIHXWWSANSR-UHFFFAOYSA-N 0.000 description 2
210000000056 organ Anatomy 0.000 description 2
238000012261 overproduction Methods 0.000 description 2
230000001575 pathological effect Effects 0.000 description 2
230000002093 peripheral effect Effects 0.000 description 2
230000001105 regulatory effect Effects 0.000 description 2
230000004044 response Effects 0.000 description 2
230000028327 secretion Effects 0.000 description 2
210000004989 spleen cell Anatomy 0.000 description 2
210000004988 splenocyte Anatomy 0.000 description 2
230000001225 therapeutic effect Effects 0.000 description 2
238000013518 transcription Methods 0.000 description 2
230000035897 transcription Effects 0.000 description 2
241000701161 unidentified adenovirus Species 0.000 description 2
239000013603 viral vector Substances 0.000 description 2
QAPSNMNOIOSXSQ-YNEHKIRRSA-N 1-[(2r,4s,5r)-4-[tert-butyl(dimethyl)silyl]oxy-5-(hydroxymethyl)oxolan-2-yl]-5-methylpyrimidine-2,4-dione Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O[Si](C)(C)C(C)(C)C)C1 QAPSNMNOIOSXSQ-YNEHKIRRSA-N 0.000 description 1
OSWFIVFLDKOXQC-UHFFFAOYSA-N 4-(3-methoxyphenyl)aniline Chemical compound COC1=CC=CC(C=2C=CC(N)=CC=2)=C1 OSWFIVFLDKOXQC-UHFFFAOYSA-N 0.000 description 1
239000013607 AAV vector Substances 0.000 description 1
102000002659 Amyloid Precursor Protein Secretases Human genes 0.000 description 1
108010043324 Amyloid Precursor Protein Secretases Proteins 0.000 description 1
241000283690 Bos taurus Species 0.000 description 1
101150044789 Cap gene Proteins 0.000 description 1
108020004705 Codon Proteins 0.000 description 1
238000007400 DNA extraction Methods 0.000 description 1
230000003682 DNA packaging effect Effects 0.000 description 1
101150029662 E1 gene Proteins 0.000 description 1
241000282326 Felis catus Species 0.000 description 1
108700039691 Genetic Promoter Regions Proteins 0.000 description 1
102100039289 Glial fibrillary acidic protein Human genes 0.000 description 1
101710193519 Glial fibrillary acidic protein Proteins 0.000 description 1
108010043121 Green Fluorescent Proteins Proteins 0.000 description 1
102000004144 Green Fluorescent Proteins Human genes 0.000 description 1
101001046686 Homo sapiens Integrin alpha-M Proteins 0.000 description 1
102100021244 Integral membrane protein GPR180 Human genes 0.000 description 1
102100022338 Integrin alpha-M Human genes 0.000 description 1
239000012097 Lipofectamine 2000 Substances 0.000 description 1
206010067125 Liver injury Diseases 0.000 description 1
241000124008 Mammalia Species 0.000 description 1
239000000020 Nitrocellulose Substances 0.000 description 1
241000283973 Oryctolagus cuniculus Species 0.000 description 1
102000004316 Oxidoreductases Human genes 0.000 description 1
241000701945 Parvoviridae Species 0.000 description 1
241000009328 Perro Species 0.000 description 1
ISWSIDIOOBJBQZ-UHFFFAOYSA-N Phenol Chemical compound OC1=CC=CC=C1 ISWSIDIOOBJBQZ-UHFFFAOYSA-N 0.000 description 1
241000288906 Primates Species 0.000 description 1
101710150114 Protein rep Proteins 0.000 description 1
101710152114 Replication protein Proteins 0.000 description 1
241000283984 Rodentia Species 0.000 description 1
108091081021 Sense strand Proteins 0.000 description 1
230000006044 T cell activation Effects 0.000 description 1
108091008874 T cell receptors Proteins 0.000 description 1
102000016266 T-Cell Antigen Receptors Human genes 0.000 description 1
210000001744 T-lymphocyte Anatomy 0.000 description 1
239000007983 Tris buffer Substances 0.000 description 1
JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 1
238000009825 accumulation Methods 0.000 description 1
230000009471 action Effects 0.000 description 1
210000001642 activated microglia Anatomy 0.000 description 1
239000002671 adjuvant Substances 0.000 description 1
239000011543 agarose gel Substances 0.000 description 1
230000032683 aging Effects 0.000 description 1
238000000137 annealing Methods 0.000 description 1
230000008901 benefit Effects 0.000 description 1
230000008499 blood brain barrier function Effects 0.000 description 1
210000001218 blood-brain barrier Anatomy 0.000 description 1
229910000389 calcium phosphate Inorganic materials 0.000 description 1
239000001506 calcium phosphate Substances 0.000 description 1
235000011010 calcium phosphates Nutrition 0.000 description 1
239000002775 capsule Substances 0.000 description 1
230000015556 catabolic process Effects 0.000 description 1
238000004113 cell culture Methods 0.000 description 1
239000013592 cell lysate Substances 0.000 description 1
230000006037 cell lysis Effects 0.000 description 1
239000002771 cell marker Substances 0.000 description 1
230000000052 comparative effect Effects 0.000 description 1
239000012228 culture supernatant Substances 0.000 description 1
230000003247 decreasing effect Effects 0.000 description 1
238000006731 degradation reaction Methods 0.000 description 1
238000007865 diluting Methods 0.000 description 1
239000003085 diluting agent Substances 0.000 description 1
201000010099 disease Diseases 0.000 description 1
208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
238000002224 dissection Methods 0.000 description 1
239000003937 drug carrier Substances 0.000 description 1
210000002919 epithelial cell Anatomy 0.000 description 1
ZMMJGEGLRURXTF-UHFFFAOYSA-N ethidium bromide Chemical compound [Br-].C12=CC(N)=CC=C2C2=CC=C(N)C=C2[N+](CC)=C1C1=CC=CC=C1 ZMMJGEGLRURXTF-UHFFFAOYSA-N 0.000 description 1
229960005542 ethidium bromide Drugs 0.000 description 1
230000005284 excitation Effects 0.000 description 1
239000013604 expression vector Substances 0.000 description 1
235000013861 fat-free Nutrition 0.000 description 1
235000019253 formic acid Nutrition 0.000 description 1
210000001652 frontal lobe Anatomy 0.000 description 1
230000004927 fusion Effects 0.000 description 1
210000004051 gastric juice Anatomy 0.000 description 1
239000000499 gel Substances 0.000 description 1
210000005046 glial fibrillary acidic protein Anatomy 0.000 description 1
210000002216 heart Anatomy 0.000 description 1
231100000234 hepatic damage Toxicity 0.000 description 1
238000009396 hybridization Methods 0.000 description 1
210000000987 immune system Anatomy 0.000 description 1
230000003053 immunization Effects 0.000 description 1
238000003018 immunoassay Methods 0.000 description 1
230000006872 improvement Effects 0.000 description 1
238000001727 in vivo Methods 0.000 description 1
230000008595 infiltration Effects 0.000 description 1
238000001764 infiltration Methods 0.000 description 1
230000002401 inhibitory effect Effects 0.000 description 1
230000005764 inhibitory process Effects 0.000 description 1
210000002490 intestinal epithelial cell Anatomy 0.000 description 1
238000001361 intraarterial administration Methods 0.000 description 1
239000007928 intraperitoneal injection Substances 0.000 description 1
238000010253 intravenous injection Methods 0.000 description 1
210000004185 liver Anatomy 0.000 description 1
230000008818 liver damage Effects 0.000 description 1
210000004072 lung Anatomy 0.000 description 1
210000002751 lymph Anatomy 0.000 description 1
210000002540 macrophage Anatomy 0.000 description 1
210000004962 mammalian cell Anatomy 0.000 description 1
230000007246 mechanism Effects 0.000 description 1
239000012528 membrane Substances 0.000 description 1
210000004088 microvessel Anatomy 0.000 description 1
239000008267 milk Substances 0.000 description 1
210000004080 milk Anatomy 0.000 description 1
235000013336 milk Nutrition 0.000 description 1
230000002438 mitochondrial effect Effects 0.000 description 1
239000000178 monomer Substances 0.000 description 1
238000010172 mouse model Methods 0.000 description 1
230000035772 mutation Effects 0.000 description 1
VMGAPWLDMVPYIA-HIDZBRGKSA-N n'-amino-n-iminomethanimidamide Chemical compound N\N=C\N=N VMGAPWLDMVPYIA-HIDZBRGKSA-N 0.000 description 1
230000001537 neural effect Effects 0.000 description 1
210000002682 neurofibrillary tangle Anatomy 0.000 description 1
229920001220 nitrocellulos Polymers 0.000 description 1
238000001543 one-way ANOVA Methods 0.000 description 1
238000004806 packaging method and process Methods 0.000 description 1
230000036961 partial effect Effects 0.000 description 1
230000008506 pathogenesis Effects 0.000 description 1
231100000915 pathological change Toxicity 0.000 description 1
230000036285 pathological change Effects 0.000 description 1
210000005259 peripheral blood Anatomy 0.000 description 1
239000011886 peripheral blood Substances 0.000 description 1
239000003755 preservative agent Substances 0.000 description 1
102000004196 processed proteins & peptides Human genes 0.000 description 1
230000001902 propagating effect Effects 0.000 description 1
230000009257 reactivity Effects 0.000 description 1
BOLDJAUMGUJJKM-LSDHHAIUSA-N renifolin D Natural products CC(=C)[C@@H]1Cc2c(O)c(O)ccc2[C@H]1CC(=O)c3ccc(O)cc3O BOLDJAUMGUJJKM-LSDHHAIUSA-N 0.000 description 1
101150066583 rep gene Proteins 0.000 description 1
230000010076 replication Effects 0.000 description 1
238000002415 sodium dodecyl sulfate polyacrylamide gel electrophoresis Methods 0.000 description 1
210000000952 spleen Anatomy 0.000 description 1
238000010561 standard procedure Methods 0.000 description 1
238000010254 subcutaneous injection Methods 0.000 description 1
239000007929 subcutaneous injection Substances 0.000 description 1
KDYFGRWQOYBRFD-UHFFFAOYSA-L succinate(2-) Chemical compound [O-]C(=O)CCC([O-])=O KDYFGRWQOYBRFD-UHFFFAOYSA-L 0.000 description 1
230000002459 sustained effect Effects 0.000 description 1
210000003478 temporal lobe Anatomy 0.000 description 1
JADVWWSKYZXRGX-UHFFFAOYSA-M thioflavine T Chemical compound [Cl-].C1=CC(N(C)C)=CC=C1C1=[N+](C)C2=CC=C(C)C=C2S1 JADVWWSKYZXRGX-UHFFFAOYSA-M 0.000 description 1
230000001988 toxicity Effects 0.000 description 1
231100000419 toxicity Toxicity 0.000 description 1
230000032258 transport Effects 0.000 description 1
QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 1
LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
238000005199 ultracentrifugation Methods 0.000 description 1
210000002438 upper gastrointestinal tract Anatomy 0.000 description 1
210000003556 vascular endothelial cell Anatomy 0.000 description 1
238000001262 western blot Methods 0.000 description 1

Images

Classifications

- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/861—Adenoviral vectors
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Chemical & Material Sciences (AREA)
Engineering & Computer Science (AREA)
Organic Chemistry (AREA)
Genetics & Genomics (AREA)
Biomedical Technology (AREA)
General Health & Medical Sciences (AREA)
Zoology (AREA)
Medicinal Chemistry (AREA)
Molecular Biology (AREA)
Neurology (AREA)
Bioinformatics & Cheminformatics (AREA)
Biophysics (AREA)
Biochemistry (AREA)
Biotechnology (AREA)
Toxicology (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Public Health (AREA)
Veterinary Medicine (AREA)
Pharmacology & Pharmacy (AREA)
Wood Science & Technology (AREA)
Gastroenterology & Hepatology (AREA)
General Engineering & Computer Science (AREA)
Animal Behavior & Ethology (AREA)
Neurosurgery (AREA)
Plant Pathology (AREA)
Psychiatry (AREA)
Virology (AREA)
Physics & Mathematics (AREA)
Hospice & Palliative Care (AREA)
General Chemical & Material Sciences (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
Microbiology (AREA)
Chemical Kinetics & Catalysis (AREA)
Epidemiology (AREA)
Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Micro-Organisms Or Cultivation Processes Thereof (AREA)

JP2003371103A 2003-06-13 2003-10-30 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター Expired - Lifetime JP4888876B2 (ja)

Priority Applications (15)

Application Number	Priority Date	Filing Date	Title
JP2003371103A JP4888876B2 (ja)	2003-06-13	2003-10-30	アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
PCT/JP2004/008224 WO2004111250A1 (ja)	2003-06-13	2004-06-11	アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
AT04745818T ATE466093T1 (de)	2003-06-13	2004-06-11	Rekombinanter adeno-assoziierter virusvektor zur behandlung von morbus alzheimer
EP04745818A EP1634956B1 (en)	2003-06-13	2004-06-11	Recombinant adeno-associated virus vector for treatment of alzheimer disease
CA2529179A CA2529179C (en)	2003-06-13	2004-06-11	Recombinant adeno-associated virus vector for treatment of alzheimer disease
DE602004026867T DE602004026867D1 (de)	2003-06-13	2004-06-11	Rekombinanter adeno-assoziierter virusvektor zur behandlung von morbus alzheimer
ES04745818T ES2345151T3 (es)	2003-06-13	2004-06-11	Vector de virus adeno-asociado recombinante para el tratamiento de la enfermedad de alzheimer.
BRPI0411321-7A BRPI0411321A (pt)	2003-06-13	2004-06-11	vetor viral associado a adenovìrus, composição farmacêutica e método para tratar doença de alzheimer, e, uso do vetor viral associado a adenovìrus
RU2006101149/13A RU2335542C2 (ru)	2003-06-13	2004-06-11	Аденоассоциированный вирусный вектор для лечения болезни альцгеймера, его применение для получения терапевтических средств, а также способ лечения болезни альцгеймера с помощью данного вектора
AU2004248014A AU2004248014B2 (en)	2003-06-13	2004-06-11	Recombinant adeno-associated virus vector for treatment of Alzheimer disease
KR1020057023896A KR20060029225A (ko)	2003-06-13	2004-06-11	알츠하이머 질환 치료용 재조합 아데노-관련 바이러스 벡터
US10/560,280 US8318687B2 (en)	2003-06-13	2004-06-11	Recombinant adeno-associated virus vector for treatment of Alzheimer disease
DK04745818.7T DK1634956T3 (da)	2003-06-13	2004-06-11	Rekombinant andeno-associeret virusvektor til behandling af Alzheimers sygdom
PT04745818T PT1634956E (pt)	2003-06-13	2004-06-11	Vector de vírus recombinante adeno-associado para o tratamento da doença de alzheimer
NZ544554A NZ544554A (en)	2003-06-13	2004-06-11	Recombinant adeno-associated virus vector for expressing beta-amyloid peptide in intestinal cells to treat Alzheimer's disease

Applications Claiming Priority (3)

Application Number	Priority Date	Filing Date	Title
JP2003169714		2003-06-13
JP2003169714		2003-06-13
JP2003371103A JP4888876B2 (ja)	2003-06-13	2003-10-30	アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター

Publications (2)

Publication Number	Publication Date
JP2005021149A JP2005021149A (ja)	2005-01-27
JP4888876B2 true JP4888876B2 (ja)	2012-02-29

Family

ID=33554414

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
JP2003371103A Expired - Lifetime JP4888876B2 (ja)	2003-06-13	2003-10-30	アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター

Country Status (15)

Country	Link
US (1)	US8318687B2 (ru)
EP (1)	EP1634956B1 (ru)
JP (1)	JP4888876B2 (ru)
KR (1)	KR20060029225A (ru)
AT (1)	ATE466093T1 (ru)
AU (1)	AU2004248014B2 (ru)
BR (1)	BRPI0411321A (ru)
CA (1)	CA2529179C (ru)
DE (1)	DE602004026867D1 (ru)
DK (1)	DK1634956T3 (ru)
ES (1)	ES2345151T3 (ru)
NZ (1)	NZ544554A (ru)
PT (1)	PT1634956E (ru)
RU (1)	RU2335542C2 (ru)
WO (1)	WO2004111250A1 (ru)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
DE10303974A1 (de)	2003-01-31	2004-08-05	Abbott Gmbh & Co. Kg	Amyloid-β(1-42)-Oligomere, Verfahren zu deren Herstellung und deren Verwendung
TWI349001B (en)	2005-03-18	2011-09-21	Lg Chemical Ltd	Method of producing unsaturated acid from olefin
WO2006112553A2 (en) *	2005-04-20	2006-10-26	Dnavec Corporation	Highly safe intranasally administrable gene vaccines for treating alzheimer's disease
CN101228272A (zh) *	2005-04-20	2008-07-23	生物载体株式会社	用于治疗阿尔茨海默病的具有较高安全性的经鼻腔内给药基因疫苗
WO2006126682A1 (ja) *	2005-05-27	2006-11-30	Juridical Foundation The Chemo-Sero-Therapeutic Research Institute	アルツハイマー病の予防・治療用ワクチン
DK2302070T3 (da) *	2005-06-23	2012-11-26	Keygene Nv	Strategier til identifikation og detektion af polymorfismer med højt gennemløb
US10316364B2 (en)	2005-09-29	2019-06-11	Keygene N.V.	Method for identifying the source of an amplicon
WO2007037678A2 (en)	2005-09-29	2007-04-05	Keygene N.V.	High throughput screening of mutagenized populations
HRP20140240T4 (hr)	2005-11-30	2017-02-24	Abbvie Inc.	Monoklonalna antitijela protiv amiloidnih beta proteina i njihova upotreba
US8691224B2 (en)	2005-11-30	2014-04-08	Abbvie Inc.	Anti-Aβ globulomer 5F7 antibodies
CN103937899B (zh)	2005-12-22	2017-09-08	凯津公司	用于基于aflp的高通量多态性检测的方法
EP3239304B1 (en)	2006-04-04	2020-08-19	Keygene N.V.	High throughput detection of molecular markers based on aflp and high troughput sequencing
US8455626B2 (en)	2006-11-30	2013-06-04	Abbott Laboratories	Aβ conformer selective anti-aβ globulomer monoclonal antibodies
US8895004B2 (en)	2007-02-27	2014-11-25	AbbVie Deutschland GmbH & Co. KG	Method for the treatment of amyloidoses
US8673846B2 (en)	2009-11-02	2014-03-18	Toagosei Co. Ltd.	Cell proliferation-promoting peptide and use thereof
US8945576B2 (en)	2010-01-08	2015-02-03	Kyoto University	Vaccine for treatment of tautopathy
US8987419B2 (en)	2010-04-15	2015-03-24	AbbVie Deutschland GmbH & Co. KG	Amyloid-beta binding proteins
US8822408B2 (en)	2010-06-04	2014-09-02	Toagosei Co., Ltd.	Cell growth-promoting peptide and use thereof
US9238796B2 (en)	2010-06-04	2016-01-19	Toagosei Co. Ltd.	Cell growth-promoting peptide and use thereof
EP3533803B1 (en)	2010-08-14	2021-10-27	AbbVie Inc.	Anti-amyloid-beta antibodies
NZ701693A (en)	2012-04-18	2017-02-24	The Children’S Hospital Of Philadelphia	Composition and methods for highly efficient gene transfer using aav capsid variants
WO2013180011A1 (ja)	2012-05-28	2013-12-05	東亞合成株式会社	抗菌ペプチド及びその利用
JP6311935B2 (ja)	2012-10-18	2018-04-18	東亞合成株式会社	２型ｔｎｆ受容体の発現を抑制する合成ペプチド及びその利用
US10195257B2 (en)	2013-07-28	2019-02-05	Qantu Therapeutics, Inc.	Vaccine formulations comprising quillaja desacylsaponins and beta amyloid peptides or tau protein to induce a Th2 immune response
EP3151866B1 (en)	2014-06-09	2023-03-08	Voyager Therapeutics, Inc.	Chimeric capsids
RU2716991C2 (ru)	2014-11-05	2020-03-17	Вояджер Терапьютикс, Инк.	Полинуклеотиды aadc для лечения болезни паркинсона
KR20230145206A (ko)	2014-11-14	2023-10-17	보이저 테라퓨틱스, 인크.	조절성 폴리뉴클레오티드
WO2016077687A1 (en)	2014-11-14	2016-05-19	Voyager Therapeutics, Inc.	Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en)	2014-12-12	2023-07-11	Voyager Therapeutics, Inc.	Compositions and methods for the production of scAAV
EP3384035A4 (en)	2015-12-02	2019-08-07	Voyager Therapeutics, Inc.	ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES
EP3448874A4 (en)	2016-04-29	2020-04-22	Voyager Therapeutics, Inc.	COMPOSITIONS FOR TREATING A DISEASE
US11299751B2 (en)	2016-04-29	2022-04-12	Voyager Therapeutics, Inc.	Compositions for the treatment of disease
KR102427379B1 (ko)	2016-05-18	2022-08-02	보이저 테라퓨틱스, 인크.	헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko)	2016-05-18	2024-04-30	보이저 테라퓨틱스, 인크.	조절성 폴리뉴클레오티드
WO2018044933A1 (en)	2016-08-30	2018-03-08	The Regents Of The University Of California	Methods for biomedical targeting and delivery and devices and systems for practicing the same
CA3048313A1 (en)	2017-01-06	2018-07-12	Stabilitech Biopharma Ltd	Virus
JP2020518258A (ja)	2017-05-05	2020-06-25	ボイジャーセラピューティクスインコーポレイテッドＶｏｙａｇｅｒＴｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ．	筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JP2020518259A (ja)	2017-05-05	2020-06-25	ボイジャーセラピューティクスインコーポレイテッドＶｏｙａｇｅｒＴｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ．	ハンチントン病治療組成物および方法
JOP20190269A1 (ar)	2017-06-15	2019-11-20	Voyager Therapeutics Inc	بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en)	2017-07-17	2019-01-24	Voyager Therapeutics, Inc.	Trajectory array guide system
CN111201030B (zh)	2017-07-25	2024-11-01	真和制药有限公司	通过阻断tim-3和其配体的相互作用治疗癌症
EP3808849A1 (en)	2017-08-03	2021-04-21	Voyager Therapeutics, Inc.	Compositions and methods for delivery of aav
AU2018338728B2 (en)	2017-09-29	2025-01-02	Centre National De La Recherche Scientifique (Cnrs)	Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
TW202413649A (zh)	2017-10-16	2024-04-01	美商航海家醫療公司	肌萎縮性脊髓側索硬化症(als)之治療
EP3697908A1 (en)	2017-10-16	2020-08-26	Voyager Therapeutics, Inc.	Treatment of amyotrophic lateral sclerosis (als)
KR20210019996A (ko)	2018-05-15	2021-02-23	보이저 테라퓨틱스, 인크.	파킨슨병의 치료를 위한 조성물 및 방법
WO2020030954A1 (en)	2018-08-09	2020-02-13	Integrative Medicine Clinic, Sia	Theranostics-like protein sanps conjugated to integrin and pmsa targeting peptides and therapy of prostate cancer
EP3856762A1 (en)	2018-09-28	2021-08-04	Voyager Therapeutics, Inc.	Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020160156A2 (en)	2019-01-30	2020-08-06	Immutics, Inc.	Anti-gal3 antibodies and uses thereof
EP4157338A4 (en)	2020-05-26	2024-11-13	TrueBinding, Inc.	METHOD FOR TREATING INFLAMMATORY DISEASES BY GALECTIN-3 BLOCKING
WO2022026410A2 (en)	2020-07-27	2022-02-03	Voyager Therapeutics, Inc	Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt)	2020-07-27	2023-04-11	Voyager Therapeutics Inc	Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
US20240050524A1 (en) *	2020-12-18	2024-02-15	Baylor College Of Medicine	Delivery of abeta variants for aggregation inhibition
US20250049955A1 (en)	2021-11-17	2025-02-13	Voyager Therapeutics, Inc.	Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
AU2023427408A1 (en)	2023-02-02	2025-09-04	Voyager Therapeutics, Inc.	Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
US5854204A (en) *	1995-03-14	1998-12-29	Praecis Pharmaceuticals, Inc.	Aβ peptides that modulate β-amyloid aggregation
US5858351A (en)	1996-01-18	1999-01-12	Avigen, Inc.	Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
TWI239847B (en) *	1997-12-02	2005-09-21	Elan Pharm Inc	N-terminal fragment of Abeta peptide and an adjuvant for preventing and treating amyloidogenic disease
US7964192B1 (en) *	1997-12-02	2011-06-21	Janssen Alzheimer Immunotherapy	Prevention and treatment of amyloidgenic disease
AU2486900A (en) *	1998-12-29	2000-07-31	University Of Georgia Research Foundation, Inc., The	Rubredoxin fusion proteins, protein expression system and methods
EP1204674A4 (en) *	1999-07-27	2005-06-01	Abgenix Inc	METHODS AND COMPOUNDS FOR PREVENTING POLYPEPTIDE COLLECTIONS ASSOCIATED WITH NEUROLOGICAL DISORDERS
US20030165481A1 (en) *	2000-02-24	2003-09-04	Hersh Louis B.	Amyloid peptide inactivating enzyme to treat Alzheimer's disease
EP1538163A3 (en) *	2000-11-01	2005-06-15	Insight Biotechnology Limited	Phosphorylated Amyloid-Beta 1-43 Protein and its use in the treatment of Alzheimer's disease
US6906169B2 (en) *	2001-05-25	2005-06-14	United Biomedical, Inc.	Immunogenic peptide composition comprising measles virus Fprotein Thelper cell epitope (MUFThl-16) and N-terminus of β-amyloid peptide
CN100450551C (zh)	2002-11-29	2009-01-14	中国医学科学院基础医学研究所	用于治疗和预防阿尔茨海默病的重组腺相关病毒基因疫苗及其用途

2003
- 2003-10-30 JP JP2003371103A patent/JP4888876B2/ja not_active Expired - Lifetime
2004
- 2004-06-11 DK DK04745818.7T patent/DK1634956T3/da active
- 2004-06-11 RU RU2006101149/13A patent/RU2335542C2/ru not_active IP Right Cessation
- 2004-06-11 WO PCT/JP2004/008224 patent/WO2004111250A1/ja not_active Ceased
- 2004-06-11 US US10/560,280 patent/US8318687B2/en not_active Expired - Fee Related
- 2004-06-11 ES ES04745818T patent/ES2345151T3/es not_active Expired - Lifetime
- 2004-06-11 CA CA2529179A patent/CA2529179C/en not_active Expired - Fee Related
- 2004-06-11 DE DE602004026867T patent/DE602004026867D1/de not_active Expired - Lifetime
- 2004-06-11 NZ NZ544554A patent/NZ544554A/en unknown
- 2004-06-11 KR KR1020057023896A patent/KR20060029225A/ko not_active Ceased
- 2004-06-11 EP EP04745818A patent/EP1634956B1/en not_active Expired - Lifetime
- 2004-06-11 BR BRPI0411321-7A patent/BRPI0411321A/pt not_active IP Right Cessation
- 2004-06-11 AT AT04745818T patent/ATE466093T1/de not_active IP Right Cessation
- 2004-06-11 PT PT04745818T patent/PT1634956E/pt unknown
- 2004-06-11 AU AU2004248014A patent/AU2004248014B2/en not_active Ceased

Also Published As

Publication number	Publication date
ES2345151T3 (es)	2010-09-16
BRPI0411321A (pt)	2006-07-18
AU2004248014A1 (en)	2004-12-23
NZ544554A (en)	2010-06-25
DK1634956T3 (da)	2010-08-16
AU2004248014B2 (en)	2008-12-04
PT1634956E (pt)	2010-06-23
US8318687B2 (en)	2012-11-27
CA2529179A1 (en)	2004-12-23
EP1634956A1 (en)	2006-03-15
RU2006101149A (ru)	2006-06-27
US20090004144A1 (en)	2009-01-01
ATE466093T1 (de)	2010-05-15
EP1634956B1 (en)	2010-04-28
EP1634956A4 (en)	2007-01-24
DE602004026867D1 (de)	2010-06-10
RU2335542C2 (ru)	2008-10-10
KR20060029225A (ko)	2006-04-05
JP2005021149A (ja)	2005-01-27
WO2004111250A1 (ja)	2004-12-23
CA2529179C (en)	2012-08-21

Legal Events

Date	Code	Title	Description
2005-12-14	A711	Notification of change in applicant	Free format text: JAPANESE INTERMEDIATE CODE: A712 Effective date: 20051213
2006-05-30	A621	Written request for application examination	Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20060529
2006-10-17	A521	Request for written amendment filed	Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20061016
2009-08-24	A131	Notification of reasons for refusal	Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20090821
2009-10-20	A601	Written request for extension of time	Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20091019
2009-10-23	A602	Written permission of extension of time	Free format text: JAPANESE INTERMEDIATE CODE: A602 Effective date: 20091022
2009-11-20	A521	Request for written amendment filed	Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20091119
2010-05-15	A711	Notification of change in applicant	Free format text: JAPANESE INTERMEDIATE CODE: A712 Effective date: 20100423
2010-08-06	A711	Notification of change in applicant	Free format text: JAPANESE INTERMEDIATE CODE: A712 Effective date: 20100712
2010-11-30	A02	Decision of refusal	Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20101130
2011-01-27	A711	Notification of change in applicant	Free format text: JAPANESE INTERMEDIATE CODE: A711 Effective date: 20110126
2011-02-15	A521	Request for written amendment filed	Free format text: JAPANESE INTERMEDIATE CODE: A821 Effective date: 20110126
2011-02-22	A711	Notification of change in applicant	Free format text: JAPANESE INTERMEDIATE CODE: A711 Effective date: 20110221
2011-03-01	A521	Request for written amendment filed	Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20110228
2011-03-10	A521	Request for written amendment filed	Free format text: JAPANESE INTERMEDIATE CODE: A821 Effective date: 20110221
2011-05-02	A911	Transfer to examiner for re-examination before appeal (zenchi)	Free format text: JAPANESE INTERMEDIATE CODE: A911 Effective date: 20110428
2011-07-25	A131	Notification of reasons for refusal	Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20110722
2011-10-08	A521	Request for written amendment filed	Free format text: JAPANESE INTERMEDIATE CODE: A821 Effective date: 20110920
2011-11-04	TRDD	Decision of grant or rejection written
2011-11-09	A01	Written decision to grant a patent or to grant a registration (utility model)	Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20111108
2011-11-10	A01	Written decision to grant a patent or to grant a registration (utility model)	Free format text: JAPANESE INTERMEDIATE CODE: A01
2011-12-15	A61	First payment of annual fees (during grant procedure)	Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20111207
2011-12-22	R150	Certificate of patent or registration of utility model	Ref document number: 4888876 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 Free format text: JAPANESE INTERMEDIATE CODE: R150
2011-12-26	FPAY	Renewal fee payment (event date is renewal date of database)	Free format text: PAYMENT UNTIL: 20141222 Year of fee payment: 3
2014-12-09	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2016-01-05	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2016-12-27	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2017-12-19	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2018-12-18	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2019-12-17	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2020-11-19	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2021-11-29	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2022-12-06	R250	Receipt of annual fees	Free format text: JAPANESE INTERMEDIATE CODE: R250
2023-10-30	EXPY	Cancellation because of completion of term

Publication	Publication Date	Title
JP4888876B2 (ja)	2012-02-29	アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
US11365229B2 (en)	2022-06-21	Methods of treating neurological diseases
US20230227802A1 (en)	2023-07-20	Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CN105408352A (zh)	2016-03-16	通过双重aav载体有效递送大基因
US20240327477A1 (en)	2024-10-03	Compositions and methods for the treatment of tdp-43 proteinopathies
CN114848850A (zh)	2022-08-05	Rs1基因在制备xlrs治疗剂中的应用及治疗剂
CN113564187A (zh)	2021-10-29	基于aav的抗血管生成基因递送系统及其用途
CN1504240A (zh)	2004-06-16	用于治疗和预防阿尔茨海默病的重组腺相关病毒基因疫苗及其用途
CN113480615B (zh)	2022-05-31	高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430B (zh)	2022-05-31	用于治疗眼部疾病的基因递送系统及其应用
CN113584043B (zh)	2022-10-11	用于治疗视网膜疾病和癌症的转基因表达盒
WO2023169115A1 (zh)	2023-09-14	一种神经系统高亲和性的aav载体及其应用
US20230129938A1 (en)	2023-04-27	Methods of treating neurological diseases
TW202235618A (zh)	2022-09-16	治療眼內壓相關疾患
US20200172590A1 (en)	2020-06-04	Methods of treating neurological diseases
CN121311586A (zh)	2026-01-09	耳蜗內毛细胞特异性启动子及其应用
JP2003146909A (ja)	2003-05-21	治療用ポリペプチドに対する免疫寛容の誘導
AU2023427408A1 (en)	2025-09-04	Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2026087567A1 (en)	2026-04-30	Transcriptional regulatory elements for cardiac-specific gene expression
Su et al.	2016	Application to Gene Therapy and Vaccination